Ravindra Singh

Scientists use a small molecule to coax synthesis of a needed protein in SMA-affected cells

posted on July 31, 2009 - 10:25am

Scientists at three U.S. institutions have used a very small synthetic molecule to correct the genetic defect in cells taken from a person with spinal muscular atrophy (SMA), a disease in which muscle-controlling nerve cells in the spinal cord are lost.